This trial will assess the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of childhood cerebral adrenoleukodystrophy (CCALD). A subject's blood stem cells will be collected and modified using the Lenti-D lentiviral vector to add a functional copy of the human ABCD1 (ATP-binding cassette, sub-family D, member 1) complementary DNA (cDNA). After modification with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.

Assessment of the proportion of subjects who have no Major Functional Disabilities (MFDs) as determined by key measures in the Neurological Function Score (NFS). [ Time Frame: 24 months (±2 months) post-transplant ] [ Designated as safety issue: No ]

Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. (Informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements).

Boys aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.

Any clinically significant cardiovascular or pulmonary, or other disease or condition that would be contraindicated for any of the other study procedures.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01896102

Locations

United States, California

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center

Los Angeles, California, United States, 90095

United States, Massachusetts

Boston Children's Hospital/Massachusetts General Hospital

Boston, Massachusetts, United States, 02115

United States, Minnesota

University of Minnesota

Minneapolis, Minnesota, United States, 55455

Sponsors and Collaborators

bluebird bio

Investigators

Study Director:

Asif Paker, M.D.

bluebird bio, Inc.

Principal Investigator:

David Williams, M.D.

Boston Children’s Hospital

Principal Investigator:

Christine Duncan, M.D.

Boston Children’s Hospital

Principal Investigator:

Florian Eichler, M.D.

Massachusetts General Hospital

Principal Investigator:

Ami J Shah, MD

University of California, Los Angeles

Principal Investigator:

Paul Orchard, MD

University of Minnesota - Clinical and Translational Science Institute